NI-0501: A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Hemophagocytic Lymphohistiocytosis

Benefits

Study subjects may or may not receive direct benefit from participating in this study. There maybe an improved chance of successfully treating your child’s HLH. Information learned in this studymay benefit other patients with HLH in the future. Six patients suffering from primary HLH havebeen treated so far with NI-0501. Of these, 5 are already scheduled for or have undergone transplant.

Treatment Period

The treatment regimen is divided into two phases: • Treatment Period 1 is characterized by close monitoring during NI-0501 administrations, 6 infusions given every 3 days. In addition, patients will receive Dexamethasone for the duration of the treatment with the study therapy. • Treatment Period 2 is characterized by less intense monitoring and includes 14 infusions,or less depending on the patient’s needs and conditions (Weeks 3 to 8). The time between NI- 0501 infusions can be increased; however this will not impact the safety assessments which will occur at least every 6 days.

About

A Swiss biotech company focused on the discovery and development of antibody-based drugs for the targeted treatment of inflammatory diseases, immune-related disorders, and cancer.